Posts about advances in Phelan-McDermid syndrome research
Important Links:Updated Community Letter & FAQ from Jaguar Gene Therapy (11/5/24)Updated information is highlighted in blueJAG201 Clinical Study Record on ClinicalTrials.govJaguar's…
After a very rigorous process with several excellent candidates, we are very excited to welcome Dr. Lauren Schmitt to the PMSF…
In January 2023, PMSF launched its research grants program to fund highly motivated scientists studying Phelan-McDermid syndrome. Our goal was to…
Jaguar Gene Therapy to Initiate Inaugural Pediatric Clinical Trial Targeting Phelan-McDermid syndrome
Jaguar Gene Therapy receives approval from FDA to conduct gene therapy clinical trials in Phelan-McDermid syndrome.
We have breaking news! Jaguar Gene Therapy has received approval from the FDA to conduct a gene therapy clinical trial in Phelan-McDermid syndrome. This is a major milestone for our community and for drug development in Phelan-McDermid syndrome! This will be the first trial aiming to target genetic underpinnings of Phelan-McDermid syndrome.
A large percentage (~75-80%) of people with a Phelan-McDermid syndrome diagnosis also have a diagnosis of autism spectrum disorder. This article…
by: Ronni Blumenthal, Phelan-McDermid Syndrome Foundation CEO.Save the Date for the 2024 Phelan-McDermid Syndrome Foundation International Family ConferenceWe are so excited…
What is SHANK3? SHANK3 is a gene that is important for the development and function of the nervous system. Genes are…
